First-in-Human and Proof-of-Mechanism Study of ARC19499 Administered to Hemophilia Patients

Phase 1

Terminated

• Conditions: Hemophilia
• Interventions: Drug: ARC19499; Drug: placebo control

• Registration Number: NCT01191372
• Lead Sponsor: Baxalta now part of Shire

Brief Summary

The purpose of this study is to examine the safety, tolerability and the way the body handles various single and multiple doses of ARC19499 in patients with hemophilia.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2010-08-26
• Study First Submitted QC: 2010-08-27
• Study First Posted: 2010-08-30
• Study Start: 2010-09-15
• Primary Completion: 2011-12-28
• Study Completion: 2011-12-28
• Status Verified: 2021-05
• Last Update Submitted: 2021-05-03
• Last Update Posted: 2021-05-05

Recruitment & Eligibility

• Status: TERMINATED
• Sex: Male
• Target Recruitment: 17

Inclusion Criteria

Adult male patients ≥18 to ≤75 years of age.

• Hemophilia of any type or severity.
• Patients who are negative for hepatitis B surface antigen (HBsAg), and human immunodeficiency virus (HIV) I and II antibody tests at screening.
• Male patients who, with their partners, are willing to use 2 effective, methods of contraception (i.e., for both self and partner) throughout the study and for at least 3 months after discontinuation of study drug treatment.
• All patients must be capable of understanding and complying with the protocol and must have signed the informed consent document.

Exclusion Criteria

• Female patients;
• If on a prophylactic coagulation factor concentrate regimen, inability or unwillingness to discontinue prophylaxis during participation in this study.
• Existence of other co-existing bleeding disorder (e.g., von Willebrand Disease).
• Medical history of venous or arterial thromboembolism.
• Scheduled for elective surgical procedure during the conduct of this study.
• Use of an investigational drug within 30 days of study entry.
• Transaminase values > 3 x upper limit of normal (ULN) at time of screening.
• Haemoglobin <12.0 g/dL.
• Participants who, in the opinion of the Investigator, have a significant infection or known inflammatory process on screening.
• Participants who have a clinically relevant history or presence of respiratory, gastrointestinal, renal, hepatic, lymphatic, neurological, cardiovascular, psychiatric, musculoskeletal, genitourinary, immunological, dermatological, connective tissue diseases or disorders.
• Any medical condition the investigator believes would place the patient at increased risk as a result of participation in the study e.g. history of thromboembolic disease or stroke.
• Any medication the investigator considers may increase the risk of adverse effects during the study.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
ARC19499 Mid Dose	ARC19499	-
ARC19499 High Dose	ARC19499	-
saline for injection	placebo control	-
ARC19499 Low Dose	ARC19499	-

Primary Outcome Measures

Name	Time	Method
Pharmacokinetics (PK) of ARC19499	2 weeks	The PK profile of ARC19499 administered by single and multiple subcutaneous injections will be characterized. The bioavailability of subcutaneously injected ARC19499 relative to that of intravenously infused ARC19499 will be determined.

Secondary Outcome Measures

Name	Time	Method
Coagulation system pharmacodynamic (PD) effects of ARC19499.	2 weeks	The PD profile of ARC19499 with respect to the kinetics of thrombin generation and clot formation will be characterized.